News

Measuring the amount of gray matter in the spinal cord could help doctors track spinal muscular atrophy (SMA) over time and see how well treatments are working, according to a study that found shrinking gray matter was tied to weaker muscles and worse motor function. More research is needed…

Regulators in the U.S. and the European Union have agreed to review Biogen’s applications seeking approval of a higher dose of Spinraza (nusinersen) for all types of spinal muscular atrophy (SMA) in individuals of all ages — a regimen the therapy’s developer says may provide more effective treatment…

A 33-year-old woman with spinal muscular atrophy (SMA) type 3 became pregnant while on Spinraza (nusinersen) treatment, and subsequently gave birth to a healthy child, according to a case report. The case “helps to elucidate new approaches for future guidelines in the management of pregnancy and SMA,” the…

Nonadherence of spinal muscular atrophy (SMA) patients to their prescribed regimen of Evrysdi (risdiplam) — an oral solution generally taken once daily after a meal — is associated with higher healthcare costs, particularly in patients with type 2 disease. That’s according to a real-world study in the…

Blood levels of creatinine, a byproduct of normal muscle function, may be a potential biomarker of disease severity and treatment response in adults and adolescents with spinal muscular atrophy (SMA), a study in China shows. Creatinine levels, overall, strongly associated with disease severity across two years of follow-up, and…

Fertility issues and low sperm counts are common in men with spinal muscular atrophy (SMA), particularly in those with more severe disease, according to a study from France. Almost two-thirds (65.9%) of SMA patients had fertility disorders. These conditions were particularly common in SMA type 2 patients. “To…

A combination of a ready-to-use SMN protein and an experimental treatment targeting the SMN2 gene, which works in a similar way to Spinraza (nusinersen), eased abnormalities in motor neurons derived from a spinal muscle atrophy (SMA) patient, researchers report. The addition of the protein, raising SMN protein levels (expression), in…

Treatment with OAV101 IT, a formulation of the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) that’s delivered directly into the spinal canal, safely led to motor improvements for children with spinal muscular atrophy (SMA) type 2 who were at least 2 years old,  top-line results from the Phase 3 STEER…

Throughout 2024, SMA News Today brought our readers daily coverage of the latest clinical research and scientific breakthroughs related to spinal muscular atrophy (SMA). Here are the top 10 most-read stories we published last year, each with a brief description. We’re excited to continue serving the SMA community in…

Nearly 4 of every 5 infants with spinal muscular atrophy (SMA) type 1 born in Italy during the era of disease-modifying therapies (DMTs) are still alive, illustrating that such treatment has “radically changed the course of … type I SMA,” with “much higher survival,” according to a nationwide…